Development of a New Score Based on Image Defined Risk Factors to Standardize Surgical Risk in Neuroblastoma Resection - A SIOPEN Collaborative Study.

Background And Aims: Image Defined Risk Factors (IDRFs) assess surgical risk in neuroblastoma (NB) and guide neoadjuvant therapy. Despite chemotherapy IDRFs may persist in 70 % of cases. Several studies have suggested that not all IDRFs hold equal significance and that the presence of an IDRF does not inherently signify unresectability.

Dual specific STAT3/5 degraders effectively block acute myeloid leukemia and natural killer/T cell lymphoma.

The transcription factors STAT3, STAT5A, and STAT5B steer hematopoiesis and immunity, but their enhanced expression and activation promote acute myeloid leukemia (AML) or natural killer/T cell lymphoma (NKCL). Current therapeutic strategies focus on blocking upstream tyrosine kinases to inhibit STAT3/5, but these kinase blockers are not selective against STAT3/5 activation and frequent resistance causes relapse, emphasizing the need for targeted drugs. We evaluated the efficacy of JPX-0700 and JPX-0750 as dual STAT3/5 binding inhibitors promoting protein degradation.

Surgical versus Medical Management of Progressive Familial Intrahepatic Cholestasis-Case Compilation and Review of the Literature.

(1) Background: Progressive familial intrahepatic cholestasis (PFIC) is a rare cause of liver failure. Surgical biliary diversion (SBD) and ileal bile salt inhibitors (IBAT) can delay or prevent liver transplantation (LTX). A comparison of the two methodologies in the literature is lacking.

Beyond image defined risk factors (IDRFs): a delphi survey highlighting definition of the surgical complexity index (SCI) in neuroblastoma.

Purpose: Preoperative evaluation of Image Defined Risk Factors (IDRFs) in neuroblastoma (NB) is crucial for determining suitability for upfront resection or tumor biopsy. IDRFs do not all carry the same weighting in predicting tumor complexity and surgical risk. In this study we aimed to assess and categorize a surgical complexity (Surgical Complexity Index, SCI) in NB resection.

MUC-FIRE: Study protocol for a randomized multicenter open-label controlled trial to show that MUCous FIstula REfeeding reduces the time from enterostomy closure to full enteral feeds.

Background: After enterostomy creation, the distal bowel to the ostomy is excluded from the physiologic passage of stool, nutrient uptake, and growth of this intestinal section. Those infants frequently require long-term parenteral nutrition, continued after enterostomy reversal due to the notable diameter discrepancy of the proximal and distal bowel. Previous studies have shown that mucous fistula refeeding (MFR) results in faster weight gain in infants.

High-content drug screening in zebrafish xenografts reveals high efficacy of dual MCL-1/BCL-X inhibition against Ewing sarcoma.

Ewing sarcoma is a pediatric bone and soft tissue cancer with an urgent need for new therapies to improve disease outcome. To identify effective drugs, phenotypic drug screening has proven to be a powerful method, but achievable throughput in mouse xenografts, the preclinical Ewing sarcoma standard model, is limited. Here, we explored the use of xenografts in zebrafish for high-throughput drug screening to discover new combination therapies for Ewing sarcoma.

Blocking STAT3/5 through direct or upstream kinase targeting in leukemic cutaneous T-cell lymphoma.

Leukemic cutaneous T-cell lymphomas (L-CTCL) are lymphoproliferative disorders of skin-homing mature T-cells causing severe symptoms and high mortality through chronic inflammation, tissue destruction, and serious infections. Despite numerous genomic sequencing efforts, recurrent driver mutations have not been identified, but chromosomal losses and gains are frequent and dominant. We integrated genomic landscape analyses with innovative pharmacologic interference studies to identify key vulnerable nodes in L-CTCL.

The "chicken-leg anastomosis": Low-cost tissue-realistic simulation model for esophageal atresia training in pediatric surgery.

Introduction: Shifting the training from the operating room (OR) to simulation models has been proven effective in enhancing patient safety and reducing the learning time to achieve competency and increase the operative efficiency. Currently the field of pediatric surgery only offers few low-cost trainers for specialized training and these feature predominantly artificial and often unrealistic tissue. The aim of this study was to develop an easy access low-cost tissue-realistic simulation model for open training of esophageal atresia and to evaluate the acceptance in trainees and junior pediatric surgeons.

The Management of Asymptomatic Congenital Pulmonary Airway Malformation: Results of a European Delphi Survey.

Consensus on the optimal management of asymptomatic congenital pulmonary airway malformation (CPAM) is lacking, and comparison between studies remains difficult due to a large variety in outcome measures. We aimed to define a core outcome set (COS) for pediatric patients with an asymptomatic CPAM. An online, three-round Delphi survey was conducted in two stakeholder groups of specialized caregivers (surgeons and non-surgeons) in various European centers.

High-Resolution Transperineal Ultrasound in Anorectal Malformations-Can We Replace the Distal Colostogram?

 Anorectal malformations (ARM) affect 1 in 5,000 newborns with a wide range of defects. In the absence of a visible fistula, the diagnosis and classification of ARM require an augmented pressure distal colostogram. This procedure can be done after a diverting colostomy has been performed and implies exposing the child to radiation.

Ultrasound-guided epidural anesthesia and sedation for open transvesical Cohen ureteric reimplantation surgery in 20 consecutive children: a prospective case series and proof-of-concept study.

Background: Epidural anesthesia is usually combined with general anesthesia (GA) for children undergoing sub-umbilical surgery and GA in children is associated with a potential for respiratory events. Aiming to reduce airway manipulation and the use of GA drugs, we designed a study of transvesical Cohen ureteteric reimplantion under epidural anesthesia in sedated, spontaneously breathing children.

Methods: We enrolled 20 children (3-83 months, 6.

Low muscle volume of the anal sphincter complex: A novel prognostic factor in children with anorectal malformations?

Background/purpose: The anal sphincter complex (ASC) plays a key role in continence and is often dysfunctional in infants born with anorectal malformations (ARM). The ASC is well depicted by magnetic resonance (MR) imaging but volumetric reference data are lacking in infants. Thus, we tested the feasibility of MR based ASC volumetry, collected reference data, and compared them with cases of favorableprognosis and unfavorable prognosis (as defined by the type of ARM).

A prospective observational study on the feasibility of subumbilical laparoscopic procedures under epidural anesthesia in sedated spontaneously breathing infants with a natural airway.

Background: Laparoscopic procedures are usually performed under general anesthesia with a secured airway including endotracheal intubation or supraglottic airways.

Aims: This is a prospective study of the feasibility of subumbilical laparoscopic procedures under epidural anesthesia in sedated, spontaneous breathing infants with a natural airway.

Methods: We consecutively enrolled 20 children <3 years old with nonpalpable testes scheduled for diagnostic laparoscopy with or without an ensuing orchidopexy, inguinal revision, or Fowler-Stephens maneuver.

Is There a Role for Elective Early Upper Gastrointestinal Contrast Study in Neurologically Impaired Children following Laparoscopic Nissen Fundoplication?

Assessment of discomfort as a sign for early postoperative complications in neurologically impaired (NI) children is challenging. The necessity of early routine upper gastrointestinal (UGI) contrast studies following laparoscopic Nissen fundoplication in NI children is unclear. We aimed to evaluate the role of scheduled UGI contrast studies to identify early postoperative complications following laparoscopic Nissen fundoplication in NI children.

Systematic review of the immunological landscape of Wilms tumors.

Results of immunotherapy in childhood solid cancer have been so far, with the exception of neuroblastoma, quite disappointing. Lack of knowledge of the immune contexture of these tumors may have contributed to the failure of immunotherapies so far. Here, we systematically reviewed the literature regarding the immunology of Wilms tumor (WT), one of the most frequent pediatric solid tumors of the abdomen.

Diagnosis and management of acute appendicitis in 21 pediatric hematology and oncology patients at a tertiary care cancer center.

Acute appendicitis is a rare gastrointestinal complication of anti-cancer chemotherapy and hematopoietic stem cell transplantation. Among a cohort of 2341 hemato-oncologic patients at a pediatric tertiary care cancer center, we identified 21 patients (0.9%) with 23 episodes of acute appendicitis, based on pathological imaging of the appendix and clinical findings.

Characterization of the Hyperintense Bronchus Sign as a Fetal MRI Marker of Airway Obstruction.

Background Fetal MRI-based differential diagnosis of congenital lung malformations is difficult because of the paucity of well-described imaging markers. Purpose To characterize the hyperintense bronchus sign (HBS) in in vivo fetal MRI of congenital lung malformation cases. Materials and Methods In this retrospective two-center study, fetal MRI scans obtained in fetuses with congenital lung malformations at US (January 2002 to September 2018) were reviewed for the HBS, a tubular or branching hyperintense structure within a lung lesion on T2-weighted images.

Is fetal magnetic resonance imaging volumetry of eventrated organs in gastroschisis predictive for surgical treatment?

Background: Fetal MRI is increasingly used in congenital abdominal wall defects. In gastroschisis, the role of fetal MRI in surgical therapy is poorly understood. Currently, the type of repair is determined primarily by clinical presentation and institutional preference.

Cysto-Vaginoscopy of a 3D-Printed Cloaca Model: A Step toward Personalized Noninvasive Preoperative Assessment in Patients with Complex Anorectal Malformations.

Introduction:  For the classification of the complexity of cloacal malformations and the decision on the operative approach, an exact anatomical assessment is mandatory. To benefit from using three-dimensional (3D)-printed models in preoperative planning and training, the practicability of these models should be guaranteed. The aim of this study was to evaluate the quality and feasibility of a real-size 3D-printed cloaca model for the purpose of cysto-vaginoscopic evaluation.

Assessment of sacral ratio in patients with anorectal malformations: Can magnetic resonance imaging replace conventional radiograph?

Introduction: In patients with anorectal malformations (ARM), the sacral ratio (SR) serves as an established predictive marker for functional prognosis and is derived from conventional radiographs. More recently, magnetic resonance imaging (MRI) has emerged as a diagnostic alternative for preoperative/baseline assessment of patients with ARM. With this study we aimed to evaluate if the SR could be reliable be measured from MRI images and if it correlated to SR measurements obtained from radiographs.

Epidural versus general anesthesia for open pyloromyotomy in infants: A retrospective observational study.

Background: Thoracic epidural anesthesia for open infantile hypertrophic pyloric stenosis surgery is a controversial issue in the presence of little comparative data.

Aims: To compare this approach to general anesthesia for desaturation events (≤90% oxygen saturation) and absolute values of minimal oxygen saturation, minimal heart frequency, operating-room occupancy time, and durations of surgery in a retrospective study design.

Methods: Data were retrieved for patients with infantile hypertrophic pyloric stenosis managed by thoracic epidurals under sedation or general anesthesia with rapid sequence induction between 01/2007 and 12/2017.

Joint Attention in a Laparoscopic Simulation-Based Training: A Pilot Study on Camera Work, Gaze Behavior, and Surgical Performance in Laparoscopic Surgery.

Training in laparoscopic surgery seems to be an important aspect in gaining and maintaining professional competency. In experimental settings, camera navigation skills improved after simulation-based training, but the effect of camera work on the surgeon's performance is not well studied. The aim of this study was to investigate whether a fixed camera or an operated camera, as well as the experience of the camera operator has an effect on the performance of the surgeon.

The Effect of Pediatric Colorectal Short-Term Medical Service Trips on Self-Reported Confidence in Patient Care in Volunteers in the Home Country.

Introduction: Short-term international medical service trips (MST) are established means to access specialized medical aid in resource-limited areas. The field of pediatric colorectal surgery is a subspecialty in pediatric surgery that mainly treats anorectal malformations (ARM) and Hirschsprung disease (HD). This study aimed to investigate the impact of MST on the donors' perception of competency concomitantly to the impact on patients in the donors' home country.

Age-Dependent Presentation and Clinical Course of 1465 Patients Aged 0 to Less than 18 Years with Ovarian or Testicular Germ Cell Tumors; Data of the MAKEI 96 Protocol Revisited in the Light of Prenatal Germ Cell Biology.

Objective: To evaluate prognostic factors in pediatric patients with gonadal germ cell tumors (GCT).

Methods: Patients <18 years with ovarian and testicular GCT (respectively OGCT and TGCT) were prospectively registered according to the guidelines of MAKEI 96. After resection of the primary tumor, patients staged ≥II received risk-stratified cisplatin-based combination chemotherapy.